ADA-SCID Gene Therapy Endorsed By European Medicines Agency For Marketing Authorization.
ADA-SCID Gene Therapy Endorsed By European Medicines Agency For Marketing Authorization.
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ADA-SCID 基因疗法获得欧洲药品管理局营销授权。
作者：
S. Ylä
人用药品委员会最近建议批准逆转录病毒介导的离体基因疗法，即用人ADA互补DNA转导患者自体CD 34+细胞，用于治疗腺苷脱氨酶严重联合免疫缺陷症（ADA-SCID）。1如果得到欧盟委员会的确认，这种名为Strimvelis的产品将成为欧盟批准的第三种基因疗法。根据CHMP的意见，“Strimvelis适用于治疗ADA缺乏导致的重度联合免疫缺陷（ADA-SCID）患者，这些患者没有合适的匹配相关干细胞供体。”积极的意见包括要求建立治疗后登记和对患者进行长期随访。
The Committee for Human Medicinal Products has recently recommended marketing authorization for retrovirus-mediated ex vivo gene therapy of patients' autologous CD34+ cells transduced with human ADA complementary DNA for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).1 If confirmed by the European Commission, the product, called Strimvelis, would be the third approved gene therapy in the European Union. According to the CHMP opinion, “Strimvelis is indicated for the treatment of patients with severe combined immune deficiency due to ADA deficiency (ADA-SCID), for whom no suitable matched related stem cell donor is available.” The positive opinion includes a request to establish a post-treatment registry and long-term follow-up of the patients.